Burosumab for Fibrous Dysplasia

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Fibrous DysplasiaBurosumab - Drug
Eligibility
Any Age
All Sexes
What conditions do you have?
Select

Study Summary

This trial is testing a study drug (burosumab) to see if it helps people with FD who have low blood phosphate levels. People with FD are prone to deformities, fractures, and other problems. The study will last 48 weeks, and participants will visit the NIH 3 times. They will self-inject the drug at home 1-2 times/month.

Eligible Conditions
  • Fibrous Dysplasia

Treatment Effectiveness

Effectiveness Progress

1 of 3

Study Objectives

1 Primary · 9 Secondary · Reporting Duration: 48 weeks (adults), 50 weeks (children)

24 weeks
Proportion of subjects achieving serum phosphate levels within the target range (Z-score -1 to +2) at Week 24.
48 weeks
Change and percent change from baseline to post-baseline visits in serum phosphate, serum 1,25(OH)2D, ratio of renal tubular maximum reabsorption rate of phosphate to glomerular filtration rate (TmP/GFR).
Change and percent change in serum bone turnover markers, including procollagen 1 N-terminal propeptide (P1NP), beta crosslaps C-telopeptides (CTX), osteocalcin, and bone-specific alkaline phosphatase from baseline to 48 weeks.
Change from baseline to 48 weeks in patient reported outcomes measures: SF36, SF10, Brief Fatigue Inventory
Change in FD lesion activity using 18F-NaF PET/CT total lesion activity
Change in FD lesion histology and cell proliferation as assessed by minimally invasive bone biopsies from baseline to 48 weeks (adults with capacity to consent only) from baseline to 48 weeks
Change in functional parameters: - Muscle strength - Range-of-motion - Walking speed (9-minute walk)
Skeletal changes assessed on skeletal survey at baseline and 48 weeks
The proportion of subjects achieving serum phosphate levels within the target range (Z-score -1 to +2) at Week 48
Week 50
Adverse events and clinical safety laboratory tests for up to 4 weeks after the final burosumab dose

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Side Effects for

KRN23 XLH and ENS
67%Musculoskeletal Pain
67%Pain In Extremity
33%Viral Upper Respiratory Tract Infection
33%Gingivitis
33%Upper Respiratory Tract Infection
33%Animal Bite
33%Muscle Strain
33%Nausea
33%Vomiting
33%Influenza Like Illness
33%Ulcer
33%Seasonal Allergy
33%Incision Site Cellulitis
33%Nasopharyngitis
33%Tooth Infection
33%Incision Site Rash
33%Procedural Pain
33%Thoracic Vertebral Fracture
33%Amylase Increased
33%Arthralgia
33%Osteoarthritis
33%Acrochordon
33%Anxiety
33%Cough
33%Anaemia
33%Pain
33%Peripheral Swelling
33%Tooth Fracture
33%Headache
33%Insomnia
33%Injection Site Erythema
33%Tonsillitis
33%Adverse Drug Reaction
33%Gastroenteritis
33%Lipase Increased
33%Vitamin D Deficiency
33%Musculoskeletal Chest Pain
33%Nasal Congestion
33%Hypertension
This histogram enumerates side effects from a completed 2021 Phase 2 trial (NCT02304367) in the KRN23 XLH and ENS ARM group. Side effects include: Musculoskeletal Pain with 67%, Pain In Extremity with 67%, Viral Upper Respiratory Tract Infection with 33%, Gingivitis with 33%, Upper Respiratory Tract Infection with 33%.

Trial Design

1 Treatment Group

Treatment
1 of 1

Experimental Treatment

15 Total Participants · 1 Treatment Group

Primary Treatment: Burosumab · No Placebo Group · Phase 2

Treatment
Drug
Experimental Group · 1 Intervention: Burosumab · Intervention Types: Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Burosumab
2019
Completed Phase 3
~70

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 48 weeks (adults), 50 weeks (children)

Who is running the clinical trial?

National Institute of Dental and Craniofacial Research (NIDCR)Lead Sponsor
283 Previous Clinical Trials
807,049 Total Patients Enrolled
1 Trials studying Fibrous Dysplasia
15 Patients Enrolled for Fibrous Dysplasia
Alison M Boyce, M.D.Principal InvestigatorNational Institute of Dental and Craniofacial Research (NIDCR)
3 Previous Clinical Trials
524 Total Patients Enrolled
1 Trials studying Fibrous Dysplasia
15 Patients Enrolled for Fibrous Dysplasia

Eligibility Criteria

Age Any Age · All Participants · 10 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You have a confirmed diagnosis of fibrous dysplasia.
You are willing to comply with all study procedures and are available for the duration of the study.
Periodic abstinence is the preferred method of contraception.
Male sterilization (at least 6 months prior to screening)
Use of oral, injected or implanted hormonal methods of contraception or other forms of hormonal contraception that have comparable efficacy (failure rate <1%), for example hormone vaginal ring or transdermal hormone contraception.